by Peter Ciszewski | Apr 3, 2019
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to investigational drug tinostamustine, a potentially first-in-class alkylating deacetylase inhibiting molecule being studied in early phase clinical trials, for the treatment of...
by Peter Ciszewski | Apr 3, 2019
Becky Abbott, MPH, Manager of Treatment and Research at the National Foundation for Ectodermal Dysplasias (NFED) discuses her organization and ectodermal dysplasias (ED), a group of more than 180 disorders that affect the outer layer of tissue of the embryo...
by Peter Ciszewski | Apr 2, 2019
The U.S. Food and Drug Administration (FDA) granted Fast Track Designation for Ad-RTS-hIL-12 plus veledimex for the treatment of recurrent or progressive glioblastoma multiforme (rGBM) in adults. “Recurrent glioblastoma multiforme is an aggressive and...
by Peter Ciszewski | Apr 1, 2019
The U.S. Food and Drug Administration (FDA) granted PRM-151, a novel investigational anti-fibrotic immunomodulator, Breakthrough Therapy designation for Idiopathic Pulmonary Fibrosis (IPF). Promedior recently announced that it had reached an agreement with the FDA on...
by Peter Ciszewski | Mar 29, 2019
David Atkins, PhD, CEO of Congenica, a provider of the diagnostic decision support platform, Sapientia, which enable clinicians to interrogate the human genome to identify disease-causing variants. 80% of rare diseases are thought to have a genetic component,...